Pub Date : 2024-09-27DOI: 10.1016/j.jval.2024.08.005
Joseph Kwon, Rakhee Raghunandan, Son Hong Nghiem, Kirsten Howard, Emily Lancsar, Elisabeth Huynh, Martin Howell, Stavros Petrou, Sarah Smith
Objectives: Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.
Methods: Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.
Results: Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: "Get aches and pains" for physical functioning; "Feel sad/blue" for emotional functioning; "Other kids not friends" for social functioning; and "Keeping up with school work" for school functioning.
Conclusions: The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.
目的:儿科生活质量量表(Pediatric Quality of Life InventoryTM Version 4.0 Generic Core Scales,简称 PedsQL GCS)由 23 个项目组成,涵盖四个分量表(身体、情绪、社交和学校功能),是一种广泛应用的儿童健康相关生活质量通用测量方法,但不能为基于成本效益的决策提供健康效用。本研究旨在开发一个可用于澳大利亚健康效用推导的 PedsQL GCS 简化项目版本:数据来源于《澳大利亚儿童纵向研究》的两个队列,包括所有 PedsQL GCS 版本(幼儿、幼儿、儿童、青少年)的代理回复,以及包含儿童版本自我报告的 CheckPoint 样本。三个分析样本分别是CheckPoint 样本(n=1,874)、Mallinson 样本(n=7,855)和幼儿样本(n=7,401)。探索性和确认性因素分析对维度进行了评估。心理测量分析对每个样本随机抽取的三个子样本(样本数=500)采用了 Rasch 和经典标准。项目选择优先考虑在 CheckPoint 样本中的心理测量表现,同时也考虑在其他样本中的表现和概念内容:维度评估没有为测量结果生成其他经验结构,因此对原有的四个子量表进行了心理测量分析。选定的项目有身体机能:"疼痛";情绪机能:"悲伤/忧郁";社会功能:"其他孩子不是朋友";学校功能:"跟上学校功课":最终的四项目集(有待进一步的心理计量验证和评估)可以从广泛使用的儿童生活质量量表 GCS 中生成健康效用,为基于成本效益的决策提供依据。
{"title":"Development of a Health-State Classification System for the Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales for Preference-Based Valuation in Australia.","authors":"Joseph Kwon, Rakhee Raghunandan, Son Hong Nghiem, Kirsten Howard, Emily Lancsar, Elisabeth Huynh, Martin Howell, Stavros Petrou, Sarah Smith","doi":"10.1016/j.jval.2024.08.005","DOIUrl":"10.1016/j.jval.2024.08.005","url":null,"abstract":"<p><strong>Objectives: </strong>Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.</p><p><strong>Methods: </strong>Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.</p><p><strong>Results: </strong>Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: \"Get aches and pains\" for physical functioning; \"Feel sad/blue\" for emotional functioning; \"Other kids not friends\" for social functioning; and \"Keeping up with school work\" for school functioning.</p><p><strong>Conclusions: </strong>The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-27DOI: 10.1016/j.jval.2024.08.003
Dany Habka, William C Hsu, Joseph Antoun
Objectives: According to most guidelines, dietary interventions are essential in the management of diabetes. Fasting has emerged as potential therapeutic regimes for diabetes. The proof-of-concept study and the fasting in diabetes treatment trial are the first to explore the clinical impact of the Fasting Mimicking Diet (FMD) in patients with type 2 diabetes mellitus. Their results showed that FMD cycles improve glycemic management and can be integrated into usual care complementary to current guidelines. This economic evaluation aims to assess the 10-year quality-of-life effects, cost implications, and cost-effectiveness of adding a 3-year FMD program to diabetes standard care in diabetic population on dual or triple medications at baseline from the perspective of the US payer.
Methods: We constructed a microsimulation model in TreeAge using a published US-specific diabetes model. The model was populated using FMD effectiveness outcomes and publicly available clinical and economic data associated with diabetes complications, use of diabetes medications, hypoglycemia incidence, direct medical costs in 2021 USD, quality of life, and mortality. All benefits were discounted by 3%.
Results: This cost-utility analysis showed that the FMD program was associated with 11.4% less diabetes complications, 67.2% less overall diabetes medication use, and 45.0% less hypoglycemia events over the 10-year simulation period. The program generated an additional effectiveness benefit of 0.211 quality-adjusted life year and net monetary benefit of 41 613 USD per simulated patient. Thus, the FMD program is cost saving.
Conclusions: These results indicate that the FMD program is a beneficial first-line strategy in T2DM management.
{"title":"Economic Evaluation of Fasting Mimicking Diet vs Standard Care in Diabetic Patients on Dual or Triple Medications at Baseline in the United States: A Cost-Utility Analysis.","authors":"Dany Habka, William C Hsu, Joseph Antoun","doi":"10.1016/j.jval.2024.08.003","DOIUrl":"10.1016/j.jval.2024.08.003","url":null,"abstract":"<p><strong>Objectives: </strong>According to most guidelines, dietary interventions are essential in the management of diabetes. Fasting has emerged as potential therapeutic regimes for diabetes. The proof-of-concept study and the fasting in diabetes treatment trial are the first to explore the clinical impact of the Fasting Mimicking Diet (FMD) in patients with type 2 diabetes mellitus. Their results showed that FMD cycles improve glycemic management and can be integrated into usual care complementary to current guidelines. This economic evaluation aims to assess the 10-year quality-of-life effects, cost implications, and cost-effectiveness of adding a 3-year FMD program to diabetes standard care in diabetic population on dual or triple medications at baseline from the perspective of the US payer.</p><p><strong>Methods: </strong>We constructed a microsimulation model in TreeAge using a published US-specific diabetes model. The model was populated using FMD effectiveness outcomes and publicly available clinical and economic data associated with diabetes complications, use of diabetes medications, hypoglycemia incidence, direct medical costs in 2021 USD, quality of life, and mortality. All benefits were discounted by 3%.</p><p><strong>Results: </strong>This cost-utility analysis showed that the FMD program was associated with 11.4% less diabetes complications, 67.2% less overall diabetes medication use, and 45.0% less hypoglycemia events over the 10-year simulation period. The program generated an additional effectiveness benefit of 0.211 quality-adjusted life year and net monetary benefit of 41 613 USD per simulated patient. Thus, the FMD program is cost saving.</p><p><strong>Conclusions: </strong>These results indicate that the FMD program is a beneficial first-line strategy in T2DM management.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-04DOI: 10.1016/j.jval.2024.08.002
Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem
Objectives: This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.
Methods: A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).
Results: Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.
Conclusions: Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.
{"title":"Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice.","authors":"Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem","doi":"10.1016/j.jval.2024.08.002","DOIUrl":"10.1016/j.jval.2024.08.002","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.</p><p><strong>Methods: </strong>A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).</p><p><strong>Results: </strong>Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.</p><p><strong>Conclusions: </strong>Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142146419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-21DOI: 10.1016/j.jval.2024.08.001
Sean D. Sullivan BScPharm, PhD , Olivier J. Wouters PhD , Emma M. Cousin PharmD , Ayuri S. Kirihennedige BSc , Inmaculada Hernandez PharmD, PhD
Objectives
By September 2024, the Centers for Medicare and Medicaid Services (CMS) will publicly report the negotiated prices (Maximum Fair Prices) for the first 10 drugs selected for price negotiation. We estimate initial price offers based on net prices, statutorily defined ceilings, and comparative effectiveness data for the 10 drugs and their therapeutic alternatives.
Methods
We utilized net prices and other price benchmarks for the 10 drugs and their therapeutic alternatives. We searched for data on comparative clinical effectiveness for the primary indications. We outlined a range of plausible initial price offers based on CMS guidance and our interpretation of regulatory intent.
Results
For ibrutinib and ustekinumab, statutorily defined ceiling prices will likely determine the initial price offers. The integration of net pricing and clinical evidence from comparator branded products will inform the initial price offers for apixaban, empagliflozin, etanercept, and insulin aspart. Rivaroxaban and sacubitril/valsartan have therapeutic alternatives that are generics; therefore, CMS may apply a discount to current net prices. To achieve savings in the negotiation of dapagliflozin and sitagliptin, CMS will have to leverage additional negotiation factors because statutory defined ceilings and net prices of therapeutic alternatives are similar or higher.
Conclusions
This analysis sheds light on important price benchmarks and clinical evidence factors for the determination of the initial price offers. Although we were not able to simulate the offer and counter-offer process, our findings provide a transparent and systematic way to produce initial offers that are consistent with CMS guidance.
{"title":"Integrating Price Benchmarks and Comparative Clinical Effectiveness to Inform the Medicare Drug Price Negotiation Program","authors":"Sean D. Sullivan BScPharm, PhD , Olivier J. Wouters PhD , Emma M. Cousin PharmD , Ayuri S. Kirihennedige BSc , Inmaculada Hernandez PharmD, PhD","doi":"10.1016/j.jval.2024.08.001","DOIUrl":"10.1016/j.jval.2024.08.001","url":null,"abstract":"<div><h3>Objectives</h3><div>By September 2024, the Centers for Medicare and Medicaid Services (CMS) will publicly report the negotiated prices (Maximum Fair Prices) for the first 10 drugs selected for price negotiation. We estimate initial price offers based on net prices, statutorily defined ceilings, and comparative effectiveness data for the 10 drugs and their therapeutic alternatives.</div></div><div><h3>Methods</h3><div>We utilized net prices and other price benchmarks for the 10 drugs and their therapeutic alternatives. We searched for data on comparative clinical effectiveness for the primary indications. We outlined a range of plausible initial price offers based on CMS guidance and our interpretation of regulatory intent.</div></div><div><h3>Results</h3><div>For ibrutinib and ustekinumab, statutorily defined ceiling prices will likely determine the initial price offers. The integration of net pricing and clinical evidence from comparator branded products will inform the initial price offers for apixaban, empagliflozin, etanercept, and insulin aspart. Rivaroxaban and sacubitril/valsartan have therapeutic alternatives that are generics; therefore, CMS may apply a discount to current net prices. To achieve savings in the negotiation of dapagliflozin and sitagliptin, CMS will have to leverage additional negotiation factors because statutory defined ceilings and net prices of therapeutic alternatives are similar or higher.</div></div><div><h3>Conclusions</h3><div>This analysis sheds light on important price benchmarks and clinical evidence factors for the determination of the initial price offers. Although we were not able to simulate the offer and counter-offer process, our findings provide a transparent and systematic way to produce initial offers that are consistent with CMS guidance.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"27 10","pages":"Pages 1348-1357"},"PeriodicalIF":4.9,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142000750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-10DOI: 10.1016/j.jval.2024.07.024
Anke van Engen, Robert Krüger, Adam Parnaby, Mihai Rotaru, James Ryan, Dima Samaha, Dimitrios Tzelis
Objectives: To assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs.
Methods: The consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated.
Results: In 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England's National Institute for Health and Care Excellence scope was included to proxy remaining MS. A minimum of 280 and 720 analyses would be requested, exponentially increasing as additional outcome measures and subgroups are requested.
Conclusions: The PICO approach outlined by EUnetHTA 21 results in a significant number of analysis requests and substantial resources. Use of complementary analyses alongside evidence-based methods to derive PICOs and engaging with the health technology developer throughout the process would create a workable EU PICO that is predictable and most impactful for the EU, resulting in a timely and high-quality assessment report that is more usable at a MS level.
{"title":"The Impact of Additive Population(s), Intervention, Comparator(s), and Outcomes in a European Joint Clinical Health Technology Assessment.","authors":"Anke van Engen, Robert Krüger, Adam Parnaby, Mihai Rotaru, James Ryan, Dima Samaha, Dimitrios Tzelis","doi":"10.1016/j.jval.2024.07.024","DOIUrl":"10.1016/j.jval.2024.07.024","url":null,"abstract":"<p><strong>Objectives: </strong>To assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs.</p><p><strong>Methods: </strong>The consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated.</p><p><strong>Results: </strong>In 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England's National Institute for Health and Care Excellence scope was included to proxy remaining MS. A minimum of 280 and 720 analyses would be requested, exponentially increasing as additional outcome measures and subgroups are requested.</p><p><strong>Conclusions: </strong>The PICO approach outlined by EUnetHTA 21 results in a significant number of analysis requests and substantial resources. Use of complementary analyses alongside evidence-based methods to derive PICOs and engaging with the health technology developer throughout the process would create a workable EU PICO that is predictable and most impactful for the EU, resulting in a timely and high-quality assessment report that is more usable at a MS level.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141917572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objectives: Chronic pain is a highly debilitating condition that affects older adults and has the potential to increase their odds of experiencing cognitive impairment. The primary objective of this study was to examine the correlation between chronic pain and dementia. Additionally, this research endeavors to ascertain whether the association between chronic pain and dementia differs by age and gender.
Methods: Cross-sectional data were derived from the Survey of Disability, Ageing, and Carers. A total of 20 671 and 20 081 participants aged 65 years and older in 2015 and 2018, respectively, were included in this study. The pooled association between chronic pain and dementia was assessed using a multivariable logistic regression model. Furthermore, the study also examined the multiplicative interaction effects between chronic pain and age, as well as chronic pain and gender, with dementia.
Results: The pooled analysis demonstrated that chronic pain was associated with a heightened odds of dementia (adjusted odds ratio 1.95; 95% CI 1.85-2.05) among older Australians compared with their counterparts without chronic pain. The interaction effect indicated that individuals with chronic pain across all age groups exhibited increased odds of living with dementia. Additionally, women with chronic pain had higher odds of dementia compared with their counterparts without chronic pain and being male.
Conclusions: A continuous, coordinated, and tailored healthcare strategy is necessary to determine the pain management goals and explore early treatment options for chronic pain in older adults, particularly in groups with the greatest need.
{"title":"Age and Gender Differences in the Relationship Between Chronic Pain and Dementia Among Older Australians.","authors":"Rezwanul Haque, Khorshed Alam, Jeff Gow, Christine Neville, Syed Afroz Keramat","doi":"10.1016/j.jval.2024.07.022","DOIUrl":"10.1016/j.jval.2024.07.022","url":null,"abstract":"<p><strong>Objectives: </strong>Chronic pain is a highly debilitating condition that affects older adults and has the potential to increase their odds of experiencing cognitive impairment. The primary objective of this study was to examine the correlation between chronic pain and dementia. Additionally, this research endeavors to ascertain whether the association between chronic pain and dementia differs by age and gender.</p><p><strong>Methods: </strong>Cross-sectional data were derived from the Survey of Disability, Ageing, and Carers. A total of 20 671 and 20 081 participants aged 65 years and older in 2015 and 2018, respectively, were included in this study. The pooled association between chronic pain and dementia was assessed using a multivariable logistic regression model. Furthermore, the study also examined the multiplicative interaction effects between chronic pain and age, as well as chronic pain and gender, with dementia.</p><p><strong>Results: </strong>The pooled analysis demonstrated that chronic pain was associated with a heightened odds of dementia (adjusted odds ratio 1.95; 95% CI 1.85-2.05) among older Australians compared with their counterparts without chronic pain. The interaction effect indicated that individuals with chronic pain across all age groups exhibited increased odds of living with dementia. Additionally, women with chronic pain had higher odds of dementia compared with their counterparts without chronic pain and being male.</p><p><strong>Conclusions: </strong>A continuous, coordinated, and tailored healthcare strategy is necessary to determine the pain management goals and explore early treatment options for chronic pain in older adults, particularly in groups with the greatest need.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-09DOI: 10.1016/j.jval.2024.07.018
Brandon Lu, Erind Dvorani, Lena Nguyen, Jaclyn M Beca, Rebecca E Mercer, Andrea Adamic, Caroline Muñoz, Kelvin K W Chan
Objectives: MVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC.
Methods: We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer's perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis.
Results: The matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of -$6379 (95%CI: -9417, -3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: -0.02, 0.02) LY and -0.01 (95% CI: -0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses.
Conclusion: Bevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.
{"title":"Cost-Effectiveness Analysis of Bevacizumab Biosimilars Versus Originator Bevacizumab for Metastatic Colorectal Cancer: A Comparative Study Using Real-World Data.","authors":"Brandon Lu, Erind Dvorani, Lena Nguyen, Jaclyn M Beca, Rebecca E Mercer, Andrea Adamic, Caroline Muñoz, Kelvin K W Chan","doi":"10.1016/j.jval.2024.07.018","DOIUrl":"10.1016/j.jval.2024.07.018","url":null,"abstract":"<p><strong>Objectives: </strong>MVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC.</p><p><strong>Methods: </strong>We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer's perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis.</p><p><strong>Results: </strong>The matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of -$6379 (95%CI: -9417, -3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: -0.02, 0.02) LY and -0.01 (95% CI: -0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses.</p><p><strong>Conclusion: </strong>Bevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-09DOI: 10.1016/j.jval.2024.07.019
Lotte Steuten, Mickael Lothgren, Andrew Bruce, Marco Campioni, Adrian Towse
Objectives: Valuing and pricing the components of combination therapies can be difficult because of competition law issues, difficulty implementing different prices for the same product in alternative uses, and attributing value to each component of the combination. We propose a value attribution solution that allows all combination components to be priced according to their relative value in the combination.
Methods: We developed a value attribution solution that is universal, symmetrical, and neutral to each combination constituent, regardless of whether it is the backbone or the add-on, and complete, meaning that it will always attribute the full value of the combination between the component parts. Moreover, it can be applied to any number of components in the combination (eg, triplets or quadruplets). We compared this solution with 2 other existing approaches.
Results: The results of the proposed value attribution solution sit between those of the 2 other value attribution approaches as it combines elements of each. As the degree of additivity moves further away from one in either direction, then our general approach ratios also move, reflecting the impact of the incremental value.
Conclusions: The proposed value attribution solution for combination therapies differs from 2 existing approaches by being universally applicable and allowing for symmetry when neutral to the constituent components of the combination. To optimally contribute to policy debate and practice, various requirements for its implementation need to be well understood, including how to overcome (1) partial information, (2) whether its assumptions can be relaxed, and (3) implementation issues.
{"title":"Proposal for a General Outcome-Based Value Attribution Framework for Combination Therapies.","authors":"Lotte Steuten, Mickael Lothgren, Andrew Bruce, Marco Campioni, Adrian Towse","doi":"10.1016/j.jval.2024.07.019","DOIUrl":"10.1016/j.jval.2024.07.019","url":null,"abstract":"<p><strong>Objectives: </strong>Valuing and pricing the components of combination therapies can be difficult because of competition law issues, difficulty implementing different prices for the same product in alternative uses, and attributing value to each component of the combination. We propose a value attribution solution that allows all combination components to be priced according to their relative value in the combination.</p><p><strong>Methods: </strong>We developed a value attribution solution that is universal, symmetrical, and neutral to each combination constituent, regardless of whether it is the backbone or the add-on, and complete, meaning that it will always attribute the full value of the combination between the component parts. Moreover, it can be applied to any number of components in the combination (eg, triplets or quadruplets). We compared this solution with 2 other existing approaches.</p><p><strong>Results: </strong>The results of the proposed value attribution solution sit between those of the 2 other value attribution approaches as it combines elements of each. As the degree of additivity moves further away from one in either direction, then our general approach ratios also move, reflecting the impact of the incremental value.</p><p><strong>Conclusions: </strong>The proposed value attribution solution for combination therapies differs from 2 existing approaches by being universally applicable and allowing for symmetry when neutral to the constituent components of the combination. To optimally contribute to policy debate and practice, various requirements for its implementation need to be well understood, including how to overcome (1) partial information, (2) whether its assumptions can be relaxed, and (3) implementation issues.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-09DOI: 10.1016/j.jval.2024.07.017
George Bray, Joanna Moncrieff, Stefan Priebe, Louise Marston, Glyn Lewis, Nadia Haynes, Vanessa Pinfold, Sonia Johnson, Rachael Maree Hunter
Objectives: The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared with maintenance treatment over 24 months from mental health services, health and social care, and societal perspectives.
Methods: Nineteen mental health trusts recruited patients to the Research into Antipsychotic Discontinuation and Reduction (RADAR) randomized controlled trial. Quality-adjusted life-years were calculated from patient-reported EQ-5D-5L, with years of full capability calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources.
Results: A total of 253 participants were randomized: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in quality-adjusted life-years (-0.035; 95% CI: -0.123 to 0.052), whereas years of full capability were significantly lower in the reduction arm compared with the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective.
Conclusions: It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over 2-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.
{"title":"Cost-Utility Analysis of Antipsychotic Reduction and Discontinuation in Patients With Long-Term Schizophrenia and Psychosis in English Mental Health Trusts: The RADAR Study.","authors":"George Bray, Joanna Moncrieff, Stefan Priebe, Louise Marston, Glyn Lewis, Nadia Haynes, Vanessa Pinfold, Sonia Johnson, Rachael Maree Hunter","doi":"10.1016/j.jval.2024.07.017","DOIUrl":"10.1016/j.jval.2024.07.017","url":null,"abstract":"<p><strong>Objectives: </strong>The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared with maintenance treatment over 24 months from mental health services, health and social care, and societal perspectives.</p><p><strong>Methods: </strong>Nineteen mental health trusts recruited patients to the Research into Antipsychotic Discontinuation and Reduction (RADAR) randomized controlled trial. Quality-adjusted life-years were calculated from patient-reported EQ-5D-5L, with years of full capability calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources.</p><p><strong>Results: </strong>A total of 253 participants were randomized: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in quality-adjusted life-years (-0.035; 95% CI: -0.123 to 0.052), whereas years of full capability were significantly lower in the reduction arm compared with the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective.</p><p><strong>Conclusions: </strong>It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over 2-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}